Zobrazeno 1 - 10
of 71
pro vyhledávání: '"adeno-associated vector"'
Autor:
Nada S. Alakhras, Christopher A. Moreland, Li Chin Wong, Priyam Raut, Sid Kamalakaran, Yi Wen, Robert W. Siegel, Laurent P. Malherbe
Publikováno v:
Frontiers in Immunology, Vol 15 (2024)
Recombinant adeno-associated virus (AAV) vectors have emerged as the preferred platform for gene therapy of rare human diseases. Despite the clinical promise, host immune responses to AAV vectors and transgene remain a major barrier to the developmen
Externí odkaz:
https://doaj.org/article/5d526ac1f027445c881a5c370ba6217e
Autor:
Madison L. Sangster, Martha M. Bishop, Yizheng Yao, Jessica F. Feitor, Sanjid Shahriar, Maxwell E. Miller, Anil K. Chekuri, Bogdan Budnik, Fengfeng Bei, Yulia Grishchuk
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 2, Pp 101269- (2024)
Mucolipidosis IV (MLIV) is a rare, autosomal recessive, lysosomal disease characterized by intellectual disability, motor deficits, and progressive vision loss. Using adeno-associated vector 9 (AAV9) and AAV-PHP.B as delivery vectors, we previously d
Externí odkaz:
https://doaj.org/article/e7cd057cd76f4b5bae5aa926b9586f15
Autor:
Matthieu Drouyer, Tak-Ho Chu, Elodie Labit, Florencia Haase, Renina Gale Navarro, Deborah Nazareth, Nicole Rosin, Jessica Merjane, Suzanne Scott, Marti Cabanes-Creus, Adrian Westhaus, Erhua Zhu, Rajiv Midha, Ian E. Alexander, Jeff Biernaskie, Samantha L. Ginn, Leszek Lisowski
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 2, Pp 101234- (2024)
Gene therapies and associated technologies are transforming biomedical research and enabling novel therapeutic options for patients living with debilitating and incurable genetic disorders. The vector system based on recombinant adeno-associated vira
Externí odkaz:
https://doaj.org/article/25d01544733d4f349e805a8d768d1b0c
Autor:
Marti Cabanes-Creus, Renina Gale Navarro, Sophia H.Y. Liao, Suzanne Scott, Rodrigo Carlessi, Ramon Roca-Pinilla, Maddison Knight, Grober Baltazar, Erhua Zhu, Matthew Jones, Elena Denisenko, Alistair R.R. Forrest, Ian E. Alexander, Janina E.E. Tirnitz-Parker, Leszek Lisowski
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 28, Iss , Pp 220-237 (2023)
Recent clinical successes have intensified interest in using adeno-associated virus (AAV) vectors for therapeutic gene delivery. The liver is a key clinical target, given its critical physiological functions and involvement in a wide range of genetic
Externí odkaz:
https://doaj.org/article/12d90e3f3d744253b93a5e234204995c
Autor:
A. A. Soldatov, Zh. I. Avdeeva, D. V. Gorenkov, L. M. Khantimirova, S. G. Guseva, V. A. Merkulov
Publikováno v:
Биопрепараты: Профилактика, диагностика, лечение, Vol 22, Iss 1, Pp 6-22 (2022)
There are a lot of diseases known today, which are caused by genetic abnormalities. Advances in genetics and biotechnology brought about gene editing technologies that can produce almost any gene, which ultimately led to the emergence of a new class
Externí odkaz:
https://doaj.org/article/be78145874ca45f58a9c957d5c4e9b61
Publikováno v:
Frontiers in Bioengineering and Biotechnology, Vol 10 (2022)
Epigenetic dysregulation is an important determinant of many pathological conditions and diseases. Designer molecules that can specifically target endogenous DNA sequences provide a means to therapeutically modulate gene function. The prokaryote-deri
Externí odkaz:
https://doaj.org/article/89557838df4841e4825115c9d5a7344c
Akademický článek
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Akademický článek
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Autor:
Fábio Duarte, Mergim Ramosaj, Ed Hasanovic, Sara Regio, Melanie Sipion, Maria Rey, Nicole Déglon
Publikováno v:
Molecular therapy. Methods & clinical development, vol. 29, pp. 254-270
Mol Ther Methods Clin Dev
Mol Ther Methods Clin Dev
One obstacle to the development of gene therapies for the central nervous system is the lack of workflows for quantifying transduction efficiency in affected neural networks and ultimately predicting therapeutic potential. We integrated data from a b
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a0ea34655fa9f6ef9fbed5b989cd44f6
https://doi.org/10.1016/j.omtm.2023.03.013
https://doi.org/10.1016/j.omtm.2023.03.013
Akademický článek
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